AU2021312054A1 - Antisense oligonucleotides for RNA editing - Google Patents

Antisense oligonucleotides for RNA editing Download PDF

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Publication number
AU2021312054A1
AU2021312054A1 AU2021312054A AU2021312054A AU2021312054A1 AU 2021312054 A1 AU2021312054 A1 AU 2021312054A1 AU 2021312054 A AU2021312054 A AU 2021312054A AU 2021312054 A AU2021312054 A AU 2021312054A AU 2021312054 A1 AU2021312054 A1 AU 2021312054A1
Authority
AU
Australia
Prior art keywords
aon
editing
target
nucleotide
rna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
AU2021312054A
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English (en)
Inventor
Alicia SOLER CANTÓN
Lenka VAN SINT FIET
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
ProQR Therapeutics II BV
Original Assignee
ProQR Therapeutics II BV
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ProQR Therapeutics II BV filed Critical ProQR Therapeutics II BV
Publication of AU2021312054A1 publication Critical patent/AU2021312054A1/en
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AU2021312054A 2020-07-23 2021-07-22 Antisense oligonucleotides for RNA editing Pending AU2021312054A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB2011428.6 2020-07-23
GBGB2011428.6A GB202011428D0 (en) 2020-07-23 2020-07-23 Antisense oligonucleotides for RNA editing
PCT/EP2021/070535 WO2022018207A1 (fr) 2020-07-23 2021-07-22 Oligonucléotides antisens pour édition d'arn

Publications (1)

Publication Number Publication Date
AU2021312054A1 true AU2021312054A1 (en) 2023-03-02

Family

ID=72339468

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021312054A Pending AU2021312054A1 (en) 2020-07-23 2021-07-22 Antisense oligonucleotides for RNA editing

Country Status (7)

Country Link
US (1) US20230323346A1 (fr)
EP (1) EP4185695A1 (fr)
JP (1) JP2023535918A (fr)
AU (1) AU2021312054A1 (fr)
CA (1) CA3186366A1 (fr)
GB (1) GB202011428D0 (fr)
WO (1) WO2022018207A1 (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023152371A1 (fr) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Oligonucléotides guides pour l'édition d'acides nucléiques dans le traitement de l'hypercholestérolémie
WO2024013361A1 (fr) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucléotides pour édition d'arn médiée par adar et leur utilisation
WO2024013360A1 (fr) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3712269A1 (fr) 2014-12-17 2020-09-23 ProQR Therapeutics II B.V. Édition ciblée d'arn
JP7074345B2 (ja) 2016-06-22 2022-05-24 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 一本鎖ｒｎａ編集オリゴヌクレオチド
EP3507366B1 (fr) 2016-09-01 2020-10-07 ProQR Therapeutics II B.V. Oligonucléotides d'édition d'arn simple brin chimiquement modifiés
EP3571300A1 (fr) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Complexes oligonucléotidiques destinés à être utilisés dans l'édition d'arn
US20210079393A1 (en) 2018-02-14 2021-03-18 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US20230039928A1 (en) * 2019-12-23 2023-02-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of Stargardt disease

Also Published As

Publication number Publication date
GB202011428D0 (en) 2020-09-09
JP2023535918A (ja) 2023-08-22
EP4185695A1 (fr) 2023-05-31
US20230323346A1 (en) 2023-10-12
WO2022018207A1 (fr) 2022-01-27
CA3186366A1 (fr) 2022-01-27

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